Cardiac dysfunction in Duchenne's
Duchenne muscular dystrophy (DMD) is a severe muscle disease that causes progressive muscle weakening and degeneration.
Apr 5, 2019
0
0
Duchenne muscular dystrophy (DMD) is a severe muscle disease that causes progressive muscle weakening and degeneration.
Apr 5, 2019
0
0
'Organoids' that mimic the developing spinal cord could assist research and drug development for neurodegenerative diseases such as spinal muscular atrophy and amyotrophic lateral sclerosis.
Mar 18, 2019
0
0
In the field of muscle physiology, scientists study muscle movement at the molecular level—chiefly, proteins such as myosin, actin and titin—to understand how muscles contract.
Oct 8, 2018
0
9
A natural history study has provided the first comprehensive clinical description of spinal muscular atrophy (SMA) within the Amish and Mennonite communities and correlates ancestral chromosome 5 haplotypes and SMN2 copy ...
Sep 6, 2018
0
0
A major step forward has been made by researchers at the University of St Andrews towards finding new treatments for Spinal Muscular Atrophy (SMA), the commonest genetic cause of infant death.
Apr 18, 2018
0
4
They are many rare genetic diseases that strike perhaps only one in a million people. Often incurable, they can be profoundly debilitating and frequently life-threatening. Though each particular disease is rare, they number ...
Feb 28, 2018
0
9
A new treatment for Huntington's disease – a deadly brain disorder – has successfully completed first-in-human trials. The drug lowered levels of the harmful huntingtin protein in the spinal fluid of patients who took ...
Dec 14, 2017
0
5
In a recent paper published in the journal Skeletal Muscle, a Saint Louis University researcher reports success in identifying new drug targets that potentially could slow or halt the progression of a form muscular dystrophy, ...
Nov 7, 2017
0
251
Motor neurons are the nerves that send impulses to the muscles to generate movement. Damage of these neurons can cause very diverse diseases, for example spinal muscular atrophy in children or adult amyotrophic lateral sclerosis.
Nov 2, 2017
0
1
Scientists at the University of California, Berkeley, have engineered a new way to deliver CRISPR-Cas9 gene-editing technology inside cells and have demonstrated in mice that the technology can repair the mutation that causes ...
Oct 3, 2017
0
189